StemCell Therapy

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced positive new data across its clinical programs in Gaucher disease type 1, Fabry disease and cystinosis, further reinforcing the potential of ex vivo lentiviral gene therapy for lysosomal disorders. Additionally, AVROBIO is further expanding its lysosomal disorder pipeline with a new program in Gaucher disease type 3, which joins the recently announced program in Hunter syndrome in a synergistic portfolio of six programs designed to prevent, halt or reverse genetic disease.

Were delighted to report substantial new data across our three clinical programs. Three months post-gene therapy infusion, the first Gaucher disease patients levels of the toxic metabolite plasma lyso-Gb1, as well as plasma chitotriosidase, were lower than the baseline levels when the patient was still on enzyme replacement therapy (ERT). With our Fabry disease data continuing to reflect sustained and durable results, with our first patient now out 3.5 years from dosing, we are planning our strategy to seek accelerated approvals in one or more major markets, said Geoff MacKay, president and CEO of AVROBIO. Additionally, the first patient in the investigator-sponsored trial for cystinosis, now out one year, remains off both oral and eye drop cysteamine and we are thrilled to announce that a third patient has been dosed.

As we move into the next stage of company growth, were expanding our lysosomal disorder pipeline with a new program for Gaucher disease type 3 and we plan to dose the first Hunter syndrome patient next year. We expect to be the first lentiviral gene therapy to the clinic across all six of these indications and in some cases, the first to be in the clinic with an investigational gene therapy of any type. We believe the new data weve announced today help de-risk our portfolio which leverages the same lentiviral gene therapy approach across indications, MacKay added. With strong clinical trial enrollment momentum coming out of the COVID-19-related slowdown, we anticipate dosing, enrolling or consenting five patients across our clinical trials this quarter, and dosing a total of 30 patients cumulatively across our clinical programs by the end of 2021.

Positive clinical data out as far as 3.5 years across a broad lysosomal disorder gene therapy pipeline

New clinical updates announced today include:

At three months post-gene therapy, no unexpected safety events or trends have been identified in the trial, with no serious adverse events related to AVR-RD-02 reported in the first patient dosed as of the safety data cut-off date, Nov. 3, 2020.

The company reported durable and sustained response in enzyme activity, substrate levels and VCN across patients in both the Phase 1 and Phase 2 trials as of the data cut-off date, indicating successful engraftment of genetically modified cells and endogenous production of the functional enzyme needed to break down toxic substrate and metabolites in patients. Updated biomarker data on kidney function show generally stable estimated glomerular filtration rate (eGFR) in both Phase 1 and Phase 2 patients. Historically, people living with Fabry disease experience a progressive, faster-than-normal rate of decline in kidney function, as measured by eGFR, whether or not they are on ERT, the current standard of care. AVROBIO believes the stability in eGFR for patients in its clinical trials to be clinically significant and relevant.

No unexpected safety events or trends have been identified in the trials as of the safety data cut-off date, Oct. 8, 2020. The eight serious adverse events reported in the two Fabry disease trials have been consistent with the conditioning regimen, stem cell mobilization, underlying disease or pre-existing conditions. Pre-existing low anti-AGA antibody titers have been detected in four patients in the Fabry Phase 1 trial and a transient low titer was observed but not detectable in subsequent measures in one patient in the Fabry Phase 2 trial.

No unexpected safety events or trends have been identified in the trial, with no serious adverse events reported as of the Nov. 2, 2020, safety data cut-off date.

Pioneering approach to personalized conditioning to leverage advantages of busulfan

The company also shared new data on the safety and tolerability profile of precision conditioning with busulfan prior to gene therapy administration. AVROBIO is pioneering a new approach called targeted concentration intervention (TCI) that enables precise dosing designed to optimize engraftment durability and head-to-toe reach of ex vivo lentiviral gene therapies. TCI aims to maximize the likelihood of engraftment while minimizing the risk of out-of-range side effects by targeting busulfan exposure to an area under the curve of 90 mg x hr/L over four days, called Bu90-TCI.

In AVROBIOs clinical trials to date, data suggest that side effects from its single-agent, single-cycle approach to Bu90-TCI conditioning may be predictable, manageable and transient. The side effects have tended to be mild to moderate in nature and typically presented one week after dosing and peak over three to four days before quickly subsiding. Unlike other conditioning agents, Bu90-TCI is lymphocyte sparing, meaning that important components of the adaptive immune system, B and T cells, are expected to be minimally affected.

Strategic pipeline expansion into relentlessly progressive lysosomal disorders

AVROBIO announced the addition of Gaucher disease type 3 to its pipeline, following the recent addition of Hunter syndrome, which is planned to enter the clinic next year. Together with the existing program in Pompe disease, these make up AVROBIOs second wave of clinical programs focused on life-threatening lysosomal disorders, with the goals of preventing the central nervous system and systemic deterioration that make lysosomal disorders so devastating, normalizing lifespan and lifting the burden of chronic treatment with ERT. New preclinical data suggest that AVROBIOs proprietary tagging technology, part of its industry-leading plato gene therapy platform toolbox, further enhances the potential of its investigational gene therapies in these disorders.

We believe that all six of our pipeline programs share tremendous synergies in clinical development, manufacturing, regulatory processes and commercialization. This second wave of programs will evaluate our promising investigational therapies in diseases with high unmet medical need for patients and families, said Chris Mason, M.D., Ph.D., chief scientific officer at AVROBIO. We believe the opportunity we have to potentially prevent patients, especially children, from developing the disabilities that would otherwise result from their inherited genetic code to perhaps give them the possibility of a full and healthy life is humbling. That is our purpose; it drives all of us at AVROBIO every day.

Preclinical updates include:

End-to-end plato platform ready to enable global commercialization

AVROBIO also presented data on its industry-leading plato platform highlighting advances in chemistry, manufacturing and controls (CMC) to prepare for planned upcoming trials and potential global commercialization.

The optimized processes embedded in plato are designed to enable robust product characterization and efficient production of potent, consistent drug product on two continents, with a third site slated to become operational in Europe in 2021. New advances include:

R&D Day webcast information

A live webcast of Virtual R&D Day and accompanying slides will be available under Events and Presentations in the Investors section of the companys website at http://www.avrobio.com. An archived webcast recording of the event will be available on the website for approximately 30 days.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by its industry leading plato gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statement

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, results of preclinical studies, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, and the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, including the use of a personalized and ultra-precision busulfan conditioning regimen. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our enrollment and development timelines and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

1 Collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to UCSD from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH).

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AVROBIO Announces New Positive Clinical Data and Preclinical Data, as Well as Expanded Leading Lysosomal Disorder Gene Therapy Pipeline - Business...

People with blood cancers and rare genetic diseases in NSW may benefit from a $50 million funding boost for innovative cell and gene therapies.

Treasurer Dominic Perrottet said the funding allocated in Tuesday's budget would provide better access to cutting-edge and lifesaving treatments, including CAR T-cell therapy.

"This funding offers real hope to the families of children suffering from rare and often fatal diseases by opening the door to new, innovative therapies," Mr Perrottet said.

"This is an exciting time in medicine. There is a wave of cell and gene therapies now available for rare, previously untreatable, often fatal conditions."

Health Minister Brad Hazzard said NSW had played a leading role in global research in the development of novel cell and gene therapies to treat rare, fatal conditions.

"This investment will help ensure NSW children with these rare and life-threatening conditions are given every chance possible at a longer, happier and healthier life."

The funding will give more access to CAR T-cell therapy which modifies a person's own immune cells to attack cancer, offering hope for remission and long-term survival for children and young adults with acute lymphoblastic leukaemia and adults with diffuse large B-cell lymphoma.

It will also provide potentially sight-saving gene therapy for children with the genetic blinding eye disease, retinitis pigmentosa.

Also available would be monoclonal antibody therapy for neuroblastoma, which vastly improves the outcomes for children with that type of cancer.

The funding would also provide gene-based therapies for spinal muscular atrophy, a fatal condition with few treatment options until recently.

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NSW boost for new cell and gene therapies - Yahoo News Australia

PHILADELPHIA, Nov. 11, 2020 (GLOBE NEWSWIRE) -- Passage Bio Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today commends the newly published research of the University of Pennsylvanias (Penn) Gene Therapy Program (GTP) regarding a novel targeted approach to prevent a selective neurotoxicity seen in the sensory neurons of dorsal root ganglia (DRG) after gene therapy treatment. As previously published, this DRG toxicity has been observed after both systemic and central nervous system (CNS) delivery of gene therapy and across a variety of vectors in pre-clinical models, but clinical manifestations have not been observed.1

As part of its unique collaboration agreement with Penn, Passage Bio has certain rights to this novel DRG technology for the indications the company progresses with Penn.

Although our safety studies for our programs have not shown any clinical manifestations of DRG toxicity, we are excited about the promising approach developed by Penns GTP, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. As part of our mission to develop transformative therapies for patients, we remain committed to advancing the field of gene therapy. If in the future this new approach shows clinical benefit for patients, we will be in a strong position to incorporate it into our programs. Our relationship with Penns GTP is an important distinguishing characteristic of Passage Bio. Through our collaboration, we have ready access to world-class expertise and groundbreaking research that we can rapidly apply, if appropriate, to our therapeutic programs.

GTPs research on preventing DRG toxicity published online this week in Science Translational Medicine. According to the researchers, DRG toxicity is the result of over expression of an introduced gene, known as a transgene, in cells in the DRG, a cluster of neural cells on the outside of the spinal cord responsible for transmission of sensory messages. To correct this over expression, the GTP research team modified a transgene with a microRNA target designed to reduce the level of the transgene expression in DRG neurons as well as toxicity in DRG neurons, without affecting transduction elsewhere in the brain. That alteration eliminated more than 80 percent of the transgene expression in DRG neurons and reduced the related DRG toxicity in preclinical studies with primates.

James M. Wilson, M.D., Ph.D., director of Penns GTP and a chief scientific advisor at Passage Bio, served as a senior author of the published manuscript. Juliette Hordeaux, DVM, Ph.D., senior director of Translational Research in Penns GTP is first author. They reported that their microRNA target approach may be a straightforward way to potentially make AAV therapy for the central nervous system more safe.

As previously reported, results from preclinical toxicology studies for Passage Bios lead therapeutic programs, PBGM01 (GM1 gangliosidosis), PBKR03 (Krabbe disease), PBFT02 (FTD-GRN), were consistent with this overall AAV platform observation, and showed no clinical manifestations in detailed neurological examinations or daily observations. To proactively determine whether there is appearance of clinical signs of DRG toxicity in our clinical programs, Passage Bio will implement monitoring of patients, consisting of both nerve-conduction studies and neurological exams focused on sensory and peripheral nerve functions.

Passage Bio is advancing six programs, which include the lead programs for GM1 gangliosidosis (GM1), Krabbe disease, and frontotemporal dementia (FTD), as well as three additional programs for amyotrophic lateral sclerosis (ALS), metachromatic leukodystrophy (MLD) and Charcot-Marie-Tooth disease Type 2a (CMT2a). The company anticipates that the initial three clinical candidates will be in clinical trials in 2021. Through its collaboration agreement with Penn, Passage Bio has the option to license a total of 17 programs focused on rare, monogenic disorders of the CNS.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvanias Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with unparalleled access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at http://www.passagebio.com.

Penn Financial Disclosure

Dr. Wilson is a Penn faculty member and also a scientific collaborator, consultant and co-founder of Passage Bio. As such, he holds an equity stake in the Company, receives sponsored research funding from Passage Bio, and as an inventor of certain Penn intellectual property that is licensed to Passage Bio, he may receive additional financial benefits under the license in the future. The University of Pennsylvania also holds equity and licensing interests in Passage Bio.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including our planned IND submissions, initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators and partners ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with theSecurities and Exchange Commission(SEC), and other reports as filed with theSEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:Sarah McCabe and Zofia MitaStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.comZofia.mita@sternir.com

Media:Gwen FisherPassage Bio215-407-1548gfisher@passagebio.com

1 Juliette Hordeaux, Elizabeth L. Buza, et al. Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology, Human Gene Therapy, published online June 25, 2020.

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Passage Bio Highlights University of Pennsylvania's Gene Therapy Program's Newly Published Research to Prevent Toxicity Associated with Gene Therapy -...

DetailsCategory: DNA RNA and CellsPublished on Monday, 09 November 2020 18:03Hits: 291

NEW YORK, NY, USA I November 09, 2020 I Axovant Gene Therapies Ltd. (NASDAQ: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has lifted its clinical hold and cleared the Investigational New Drug (IND) Application to initiate a registrational study of AXO-AAV-GM2 gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease. AXO-AAV-GM2 is the first investigational gene therapy to achieve IND clearance for Tay-Sachs and Sandhoff diseases. The Company received a letter from the FDA indicating that it has satisfactorily addressed all issues related to the clinical hold.

The IND clearance of AXO-AAV-GM2, the first potentially curative treatment for Tay-Sachs and Sandhoff diseases to enter the clinic, marks a seminal moment for the entire GM2 gangliosidosis community. Along with AXO-AAV-GM1 for GM1 gangliosidosis, todays announcement represents the second IND clearance for Axovants gene therapy pipeline in the last 12 months, said Gavin Corcoran, M.D., Chief R&D Officer of Axovant. AXO-AAV-GM1 and AXO-AAV-GM2 are the first gene therapies to enter active clinical development in their indications. Each program captures the essence of our mission to liberate patients from debilitating neurological diseases by rapidly advancing these gene therapy programs into clinical development. We look forward to working with our study investigators, academic partners, the patient community, and families to begin enrollment in the study promptly.

Sue Kahn, Executive Director of the National Tay-Sachs & Allied Diseases Association (NTSAD), added, This is a big step toward a potential therapy for the GM2 community. It has been a long journey since NTSAD started supporting this promising gene therapy research in 2006. We are thrilled that it is now entering the clinic, bringing tremendous hope to families and the entire NTSAD community.

Axovant aims to advance the program through strategic partnerships with leading research organizations. The Company recently announced a partnership with Viralgen, an AskBio subsidiary, to support AAV-based vector manufacturing of clinical trial material for the registrational study. Additionally, through an existing genetic testing collaboration with Invitae, ongoing partnership with GM2 gangliosidosis patient groups, and collaboration with leading academic researchers at the University of Massachusetts Medical School and Massachusetts General Hospital, Axovant expects to begin patient identification and site startup activities in preparation for dosing children in the planned clinical study.

AXO-AAV-GM2 is an investigational gene therapy for Tay-Sachs and Sandhoff diseases, which are rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes that encode -Hexosaminidase A, HEXA and HEXB. Children affected by Tay-Sachs and Sandhoff diseases suffer from a progressively debilitating disease course and reduced life expectancy. AXO-AAV-GM2 delivers two vectors encoding the HEXA and HEXB genes directly to the central nervous system to produce a fully functional -Hexosaminidase A enzyme. In 2019, clinical evidence from two patients under an investigator-initiated study found that treatment with AXO-AAV-GM2 was generally well-tolerated and associated with improved bioactivity outcomes. In addition, the data demonstrated the attainment of normal neurodevelopmental milestones and improvement in myelination. AXO-AAV-GM2 has been granted Orphan Drug and Rare Pediatric Disease Designation by the FDA.

The study will enroll both infantile and juvenile subjects with GM2 gangliosidosis in the U.S. The two-part trial, sponsored by Axovant, will consist of (1) a dose ranging cohort evaluating the safe and efficacious dose of the gene therapy, followed by (2) an efficacy cohort, both of which form the basis of the registrational program. Terence R. Flotte, M.D., Professor of Pediatrics and Dean at the University of Massachusetts Medical School, will serve as principal investigator on the clinical trial.

About AXO-AAV-GM2

AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in theHEXA(leading to Tay-Sachs disease) orHEXB(leading to Sandhoff disease) genes that encode the two subunits of the -hexosaminidase A (HexA) enzyme. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA function by introducing a functional copy of theHEXAandHEXBgenes via delivery of two co-administered AAVrh8 vectors.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

SOURCE: Axovant Gene Therapies

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Axovant Gene Therapies Announces FDA Clearance of IND for AXO-AAV-GM2 Gene Therapy in Tay-Sachs and Sandhoff Diseases | DNA RNA and Cells | News...

PhoreMost Limited, the UK-based biopharmaceutical company and Oxford Biomedica plc (LSE:OXB) (Oxford Biomedica), a gene and cell therapy group, today announced that they have entered into a discovery collaboration to develop next-generation CAR-T cell therapies. Financial details of the agreement are not disclosed.

PhoreMost will deploy its in-house expertise and next-generation phenotypic screening platform, SITESEEKER, to identify therapeutic candidates for Oxford Biomedicas LentiVector gene therapy delivery system. The program will initially focus on CAR-T therapy and aims to develop next-generation cell therapies with significantly improved efficacy and durability.

Dr Chris Torrance, CEO, PhoreMost, said: This collaboration with Oxford Biomedica, a global pioneer in cell and gene therapies, is further recognition of the power of SITESEEKER, offering an exciting opportunity to discover and accelerate the development of clinical stage products. The natural complementarity between SITESEEKER and LentiVector offers great promise for this and future collaborations between the two companies.

John Dawson, CEO of Oxford Biomedica, said: We are excited to apply this next-generation technology to our LentiVector platform. The collaboration has the potential to deliver more effective CAR-T therapies, and we look forward to working closely with the PhoreMost team.

SITESEEKER exploits protein shape diversity to find functionally active peptides linked to any chosen disease setting, significantly enhancing the power of phenotypic screening and translation into therapeutic modalities. Based on proprietary protein interference, or PROTEINi, technology, SITESEEKER is able to systematically probe the entire proteome in a live cell environment to identify and exploit novel drug targets.

Oxford Biomedica is a world-leading pioneer of cell and gene therapies. Its LentiVector platform enables the successful development of breakthrough gene and cell-based medicines, and through collaborations with pharmaceutical partners, has delivered the first FDA and EMA approved CAR-T cell therapy.

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PhoreMost and Oxford Biomedica Enter Gene Therapy Discovery Collaboration - Technology Networks

BOSTON and LONDON, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company is scheduled to present at the Barclays Gene Editing & Gene Therapy Summit on November 16, 2020 at 11:15 AM ET.

A live webcast of the presentation will be available under "News & Events" in the Investors & Media section of the company's website at http://www.orchard-tx.com. A replay of the webcast will be archived on the Orchard website following the presentation.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

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Contacts

Investors

Renee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

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Orchard Therapeutics to Present at Barclays Gene Editing & Gene Therapy Summit - Yahoo Finance

Final Report will add the analysis of the impact of COVID-19 on this industry.

November 2020:

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Market dynamics include growth influencers, opportunities, threats, challenges, and other crucial facets

The global Cell and Gene Therapy market research report offers users with an all-inclusive package of market analysis that includes current market size, expansion rate, and value chain analysis. The global Cell and Gene Therapy market is segmented on a regional basis Europe, North America, Latin America, Asia Pacific, and Middle East & Africa as well. To offer a comprehensive view and competitive outlook of the global Cell and Gene Therapy market, our review team employs numerous methodological procedures, for instance, Porters five forces analysis.

This research report includes the analysis of various Cell and Gene Therapy market segments. The bifurcation of the global market is done based on its present and prospective inclinations. The regional bifurcation involves the present market scenario in the region along with the future projection of the global Cell and Gene Therapy market. The global market report offers an overview of expected market conditions due to changes in the technological, topographical, and economic elements.

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Increase in the Adoption of Cell and Gene Therapy to Propel the Growth of the Cell and Gene Therapy Market Between 2020-2027 - Eurowire

The global Gene Therapy market is rife with several new opportunities for market growth. This study provides a comprehensive description of the various propensities and trends that are aiding the growth of this market. The review takes a holistic approach in analyzing and decrypting the various forces of market growth. The all-inclusive review covers multiple insights on the historic trends and future projections that have shaped the current fettle of the market. It is worthwhile to note that the review is written with careful evaluation of various outliers and exceptions to market growth. In order to reconcile the exceptions, the projections made in the review leave scope for scaling and optimization.

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The global Gene Therapy Market is projected to reach a value of US$ XX Mn/Bn by the end of the forecast period. These projections are based on evidence-based research and analysis of the various parameters of market growth.

The global Gene Therapy market consists of the following key players:

The report takes a cognitive approach to decode the business strategies of each of the leading players. Besides, the report also gauges the impact of recent market developments on the growth trajectory of the global Gene Therapy market. The insights presented in the report are written after taking due cognizance of the various trends that currently exist in the industry.

The declaration of the coronavirus disease as a global health emergency, and successively a pandemic, created formidable challenges for the vendors within the global Gene Therapy market. The COVID-19 crisis caused a global economic downturn, aggravated by industrial shutdowns and supply chain disruptions. The vendors operating in the global Gene Therapy market are expected to make relentless efforts to minimize the damage caused by the pandemic over the coming years. Companies that deployed strategies of business continuity management and damage control were able to weather the storm. Therefore, COVID-19 pandemic has been a subjective crisis for various industries and players.

The report covers the following key regions:

Based on products, the Gene Therapy market can be segmented into the following categories:

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The report gives an objective and pragmatic outlook on key trends pertaining to:

The report answers, in detail, the following key questions pertaining to market expansion:

The Gene Therapy market makes projections for the following phases:

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Gene Therapy Market to Witness Marginal decline in Near Term amid COVID-19 Pandemic, Projects Fact.MR 2020 2026 - The Cloud Tribune

A new gene therapy may lessen traits of Angelman syndrome, but its makers may need to limit the dose.

By Giorgia Guglielmi, SpectrumNov. 5, 2020 , 9:30 AM

Originally published onSpectrum

A small clinical trial of a gene therapy for Angelman syndromea rare genetic condition related to autismis on hold after two participants temporarily lost the ability to walk. The safety issue is important to resolve, experts say, given that the therapy otherwise appears to be effective, and the trial could guide treatment strategies for similar brain conditions.

Biopharmaceutical companyUltragenyxin Novato, California, in collaboration with Florida-based biotech startupGeneTx, launched the trial in February to assess the safety of a therapy forAngelman syndrome, a neurodevelopmental condition characterized by intellectual disability, balance and motor problems, seizures, sleep problems and, in some cases, autism.

Angelman syndrome results from the mutation or absence of a gene calledUBE3A. People inherit two copies of UBE3A. Typically, only the maternal copy is active in neurons and the paternal copy is silent. But in people with Angelman syndrome, the maternal copy is mutated or missing, so their brain cells express no active UBE3A protein.

The drug developed by Ultragenyx and GeneTx, called GTX-102, is a short snippet of RNA called an antisense oligonucleotide thatactivates the paternal copy of UBE3Aand aims to restore the protein to typical levels. Three other companiesRoche, Biogen, and Ionisare pursuing similar therapies for the syndrome.

On 26 October, Ultragenyx and GeneTx reported that the clinical trial hadenrolled five individualswith Angelman syndrome, aged 5 to 15. The plan had been to administer to each participant a dose of GTX-102 once a month over four months. Researchers injected the drug directly into the nutrient-rich solution that envelops the brain and spinal cord through a site in the lower back.

The participants were to receive increasing doses, but all started with different amounts: Two began at the lowest dose, two started with the second-lowest dose, and one started at the second-highest dose. The final dose was about 10 times higher than the lowest dose.

After a single dose at the second-highest level, one participant developed leg weakness. The other four participants experienced the same adverse effect after taking the highest dose. The symptoms emerged one to four weeks after the participants last dose.

Two of the patients were not able to support themselves to walk and three were, but they were weaker, saysElizabeth Berry-Kravis, professor of child neurology at Rush University Medical Center in Chicago, Illinois, where the five children were treated.

The side effects appear to be a result of inflamed nerves where the drug was injected, perhaps due to accumulation of the drug in that area. In animal studies, the drug didnt cause similar adverse effects, says Ultragenyx chief executive officer,Emil Kakkis. We do know, though, that antisense oligonucleotides are known to have local toxic effects if given at high concentrations.

The participants all recovered after they received drugs that decrease inflammation, Berry-Kravis says. Even those who couldnt support themselves on their legs are walking around finethey actually are somewhat more coordinated now than they were before the study.

When the researchers evaluated the participants at day 128, all five showed significant improvements in some traits, including communication, sleep, and motor skills, Berry-Kravis says. Within weeks of the initial doses, parents and caregivers reported that the participants had acquired new words and gestures.

Were seeing things like using a fork independently for the first time ever, learning to swim on their own, using their augmentative communication device, being able to play an interactive game with the family, Berry-Kravis says. But, she adds, you cant go on with an adverse event.

Going forward, the companies plan to limit the maximum dose to a range in which the drug appears to improve traits without causing leg weakness. They also intend to change how they administer the drug so it cannot accumulate at the site of injection. The drug solution will be given to the patient with their head down to allow the drug to flow toward the brain more efficiently, Kakkis says.

Before resuming the study, the companies will seek approval from the U.S. Food and Drug Administration, says Scott Stromatt, chief medical officer of GeneTx. We hope dosing will start in the next one to two months, he says. Parents are pretty excited to resume because of the positive changes theyve observed in their children.

All drugs have a side effect at some point, saysMark Zylka, professor of cell biology and physiology at the University of North Carolina at Chapel Hill, who was not involved in the study. It seems like theyre just going to need to dial in the dosage better.

Zylka is working on atherapy for Angelman syndromethat uses the gene-editing technology CRISPR to unmute the paternal copy of UBE3A. The rapid improvement observed in the trial participants is encouraging, he says. It suggests that this idea of turning on the dads copy of the gene really has the potential to help individuals with Angelman.

Others are excited about what the trial results might mean for other brain conditions. One of the biggest questions in the field is how long the therapeutic window remains open in neurodevelopmental disorders like Angelman syndrome, saysTimothy Yu, assistant professor of pediatrics at Harvard University. The preliminary findings from the Ultragenyx and GeneTx trial suggest that the therapy can work even in teenagers.

Its still early days, and we have to be careful, Yu says. But if this result continues to hold true, thats going to be really game-changing.

This article was reprinted with permission fromSpectrum,the home of autism research news and analysis.

Go here to see the original:
Gene therapy for autism-linked condition weakened legs, robbing two people of ability to walk - Science Magazine

Stand by: A new gene therapy may lessen traits of Angelman syndrome, but its makers may need to limit the dose.

Courtesy of Ultragenyx

A small clinical trial of a gene therapy for Angelman syndrome a rare genetic condition related to autism is on hold after two participants temporarily lost the ability to walk. The safety issue is important to resolve, experts say, given that the therapy otherwise appears to be effective, and the trial could guide treatment strategies for similar brain conditions.

Biopharmaceutical company Ultragenyx in Novato, California, in collaboration with Florida-based biotech startup GeneTx, launched the trial in February to assess the safety of a therapy for Angelman syndrome, a neurodevelopmental condition characterized by intellectual disability, balance and motor problems, seizures, sleep problems and, in some cases, autism.

Angelman syndrome results from the mutation or absence of a gene called UBE3A. People inherit two copies of UBE3A. Typically, only the maternal copy is active in neurons and the paternal copy is silent. But in people with Angelman syndrome, the maternal copy is mutated or missing, so their brain cells express no active UBE3A protein.

The drug developed by Ultragenyx and GeneTx, called GTX-102, is a short snippet of RNA called an antisense oligonucleotide that activates the paternal copy of UBE3A and aims to restore the protein to typical levels. Three other companies Roche, Biogen and Ionis are pursuing similar therapies for the syndrome.

On 26 October, Ultragenyx and GeneTx reported that the clinical trial had enrolled five individuals with Angelman syndrome, aged 5 to 15. The plan had been to administer to each participant a dose of GTX-102 once a month over four months. Researchers injected the drug directly into the nutrient-rich solution that envelops the brain and spinal cord through a site in the lower back.

The participants were to receive increasing doses, but all started with different amounts: Two began at the lowest dose, two started with the second-lowest dose, and one started at the second-highest dose. The final dose was about 10 times higher than the lowest dose.

After a single dose at the second-highest level, one participant developed leg weakness. The other four participants experienced the same adverse effect after taking the highest dose. The symptoms emerged one to four weeks after the participants last dose.

Two of the patients were not able to support themselves to walk and three were, but they were weaker, says Elizabeth Berry-Kravis, professor of child neurology at Rush University Medical Center in Chicago, Illinois, where the five children were treated.

The side effects appear to be a result of inflamed nerves where the drug was injected, perhaps due to accumulation of the drug in that area. In animal studies, the drug didnt cause similar adverse effects, says Ultragenyx chief executive officer, Emil Kakkis. We do know, though, that antisense oligonucleotides are known to have local toxic effects if given at high concentrations.

The participants all recovered after they received drugs that decrease inflammation, Berry-Kravis says. Even those who couldnt support themselves on their legs are walking around fine they actually are somewhat more coordinated now than they were before the study.

When the researchers evaluated the participants at day 128, all five showed significant improvements in some traits, including communication, sleep and motor skills, Berry-Kravis says. Within weeks of the initial doses, parents and caregivers reported that the participants had acquired new words and gestures.

Were seeing things like using a fork independently for the first time ever, learning to swim on their own, using their augmentative communication device, being able to play an interactive game with the family, Berry-Kravis says. But, she adds, you cant go on with an adverse event.

Going forward, the companies plan to limit the maximum dose to a range in which the drug appears to improve traits without causing leg weakness. They also intend to change how they administer the drug so it cannot accumulate at the site of injection. The drug solution will be given to the patient with their head down to allow the drug to flow toward the brain more efficiently, Kakkis says.

Before resuming the study, the companies will seek approval from the U.S. Food and Drug Administration, says Scott Stromatt, chief medical officer of GeneTx.

We hope dosing will start in the next one to two months, he says. Parents are pretty excited to resume because of the positive changes theyve observed in their children.

All drugs have a side effect at some point, says Mark Zylka, professor of cell biology and physiology at the University of North Carolina at Chapel Hill, who was not involved in the study. It seems like theyre just going to need to dial in the dosage better.

Zylka is working on a therapy for Angelman syndrome that uses the gene-editing technology CRISPR to unmute the paternal copy of UBE3A. The rapid improvement observed in the trial participants is encouraging, he says. It suggests that this idea of turning on the dads copy of the gene really has the potential to help individuals with Angelman.

Others are excited about what the trial results might mean for other brain conditions. One of the biggest questions in the field is how long the therapeutic window remains open in neurodevelopmental disorders like Angelman syndrome, says Timothy Yu, associate professor of pediatrics at Harvard University. The preliminary findings from the Ultragenyx and GeneTx trial suggest that the therapy can work even in teenagers.

Its still early days, and we have to be careful, Yu says. But if this result continues to hold true, thats going to be really game-changing.

See the article here:
Gene therapy trial for autism-linked condition is put on hold - Spectrum

Global Hemophilia Gene Therapy Market: Overview

The global hemophilia gene therapy market is predicted to increase at a stellar pace in the years to follow. The medical fraternity has shown immense resilience in dealing with bleeding disorders, backed by the research excellence of the microbiology sector. The growing incidence of diseases related to genetic dysfunctions has out the healthcare industry on its toes. Research related to genetic mutations have given an impetus to the healthcare sector. There has been a renewed interest in genetic studies amongst researchers and scientists. The commendable results achieved from next-generation genetic studies has created a myriad of opportunities for growth within healthcare. The aforementioned factors are expected to drive sales across the global hemophilia gene therapy market.

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This review on the global hemophilia gene therapy market gives a concise description of the leading factors responsible for market expansion. The review analyses the growing relevance of gene therapies to make forecasts related to market expansion. The trends and propensities exhibited by the healthcare sector have also been enunciated herein. Henceforth, the review is an evidence-based commentary on the factors that can drive market demand over the near future. The COVID-19 crisis has had adverse impacts on several markets and industries, save the healthcare sector. Therefore, the review also points to the marked increase in revenues across this market due to the pandemic.

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Global Hemophilia Gene Therapy Market: Competitive Dynamics

Next-generation DNA sequencing and genetic mutations have played a vital role in enhancing healthcare treatments across a range of specializations. For this reason, leading players in the hemophilia gene therapy market are upping their game with genetic research. The ability of these vendors to produce visible results in patient care shall be a sound driver of market demand. A number of companies are also assisting patients in defining their own clotting factor with the help of new aids and technologies.

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Some of the market vendors have acquired visible expertise in the use of a modified virus for treatment of blood clotting. This virus is different from the one that caused the disease, enabling it to help in encoding the clotting factor. Market players can grow at the back of these advancements and distinctions over the next decade. Some of the leading vendors operating in the global hemophilia gene therapy market are Spark Therapeutics, UniQure NV, Pfizer, Inc., Ultragenyx Pharmaceutical, and Shire PLC Sangamo Therapeutics, Inc.

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Global Hemophilia Gene Therapy Market: Key Trends

The presence of a seamless industry for studying and analysing diseases related to blood clotting or bleeding has given an impetus to market growth. The next decade is expected to witness a surge in demand for hemophilia treatments. The proven excellence of gene therapy in preventing excessive bleeding in ailing individuals shall also play a vital role in market growth and maturity.

Prolonged external or internal bleeding can also be a cause of several other diseases and disorders. This has also led to increased investments in treatment of diseases related to blood clots. The next decade is expected to usher an era of advancements in gene therapy, giving a thrust to growth across the global market.

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Global Hemophilia Gene Therapy Market: Regional Landscape

On the basis of regions, the global hemophilia gene therapy market can be segmented into North America, Asia Pacific, South America, Europe, and the Middle East and Africa. The excellence of the US and Canada in gene therapy has given a thrust to the growth of the North America hemophilia gene therapy market.

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Originally posted here:
Hemophilia Gene Therapy Market: Growing prevalence of diseases related to blood clotting or bleeding to drive the market - BioSpace

As more and more gene therapies are approved, the elements that determine their success depend, increasingly, on the business decisions made early on.

There is nuance to every program but, (across-the-board) the cost of production is a big challenge, Mike Clayman, M.D., CEO and co-founder of Flexion, told BioSpace.

Flexions lead candidate, FX201 (humantakinogene hadenovec), is a case in point. This is an intra-articular gene therapy product for osteoarthritis (OA). It uses a helper-dependent adenovirus (HDAd) vector whose genetic construct the payload is a gene that expresses the interleukin-1 receptor antagonist (IL-1Ra) in the joint to control inflammation.

Inflammation is a known cause of pain, and chronic inflammation is believed to play a key role in the progression of OA, he explained.

FX201 is taken up rapidly by cells lining the joint, converting them into factories to make IL-1Ra. It quells the inflammation and then turns off the gene construct, Clayman said. It has the potential to provide long-term pain relief, functional improvement, and to actually delay the progression of the disease.

By injecting the product directly into the approximately 5 ml volume of the knee joint, he continued, The absolute amount of protein you need to create an effective intra-articular concentration is far less than that which would be needed to achieve systemic therapeutic levels. That translates into what we expect will be much lower doses of FX201, which would reduce manufacturing cost and give us flexibility to charge a price that would still be proportional to value delivered.

The product entered Phase I, a single ascending dose study, this year. Preclinical work suggests that once injected, there is no significant distribution outside the joint.

It is a non-integrating, non-replicating vector, Clayman said. There is preclinical evidence that it delivers meaningful improvement for at least one year, with the slowing of disease progression.

Company scientists believe a single injection FX201 has the potential to provide at least 6 to 12 months of pain relief and functional improvement in addition to possibly slowing disease progression, Clayman said. We believe this product profile, if realized, would represent a transformative new treatment for knee OA.

Currently, Flexion is actively pursuing the possibility of identifying sub-populations of OA patients who may differentially benefit, Clayman said.

The initial study is open broadly to OA patients to evaluate safety and tolerability. Once those data are analyzed, he said subsequent studies will consider and, if possible, incorporate biomarker, phenotypic or genotypic criteria to focus on a more restricted population.

There may be a marker perhaps a serum marker to identify a patient subset that may most benefit from the therapy, Clayman said. With 15 million patients in the U.S. with knee symptoms of OA, and 8 million annual injections to relieve knee pain, it behooves companies to identify a focal population that can most benefit.

But, he acknowledged, The genetics of OA are very complex. Its clearly a multigene disease with additional contributions from environmental and other external factors. Its not as though there are clearly defined genetic subtypes of the disease.

FX201 isnt the first OA product Flexion has developed. Zilretta (triamcinolone acetonide extended-release injectable suspension), an extended-release, intra-articular corticosteroid, was approved by the FDA in 2017 to relieve OA-related knee pain. In Phase III trials, it significantly reduced knee pain for 12 weeks and, for some patients, 16 weeks or more. Since approval, it has completed a Phase IIIb trial for synovitis.

Flexion also is developing FX301 (funapide, a selective sodium channel blocker, formulated in a thermosensitive gel) for acute post-operative pain.

This isnt a gene therapy, Clayman said. This is a peripheral nerve block that is expected to enter the clinic in 2021. The preclinical data so far suggest it may durably prevent post-op pain while preserving motor function.

The FDA and EMA are up-to-speed on gene therapy now. At Claymans meetings with the FDA, We had very robust scientific exchanges. I left impressed with the sophistication that was applied to the discussion. The EMA was similarly sophisticated, he said.

Coincidentally, the criteria for RMAT designation comparable to breakthrough designation for other products illustrating the points for clinical conditions, used OA as the example.

This indicates the FDA is thinking about creative approaches to complex diseases, Clayman said. If we have compelling data, we would not hesitate to pursue RMAT designation.

Flexion already has begun its health economics and outcomes research, modeling this therapeutic in terms of its ability to delay the need for knee replacement (which costs $25,000 to $50,000), and quality of life.

Whether payers will reimburse for gene therapies often depends as much on the price as on delivered value.

Our dose likely will give us manufacturing costs that will allow us to price FX201 much lower than a systemic therapy, which puts us in a better position, Clayman said. Of course, We still must generate compelling data.

Read more from the original source:
Bringing Flexion's OA Gene Therapy to Market Starts with Cost and Benefits in Mind - BioSpace

Known as the City of Brotherly Love, Philadelphia, Pennsylvania, now has a new monickerCellicon Valleyto honor the cell and gene therapies that companies such as Spark Therapeutics are bringing to it.

Science@Work

Nov. 6, 2020 , 2:00 PM

Affectionally named Cellicon Valley, Philadelphia is the birthplace of cell and gene therapy and home to more than 30 companies dedicated to cell and gene therapy research. Philadelphia-based Spark Therapeutics became the first to bring a U.S. Food and Drug Administration (FDA)-approved gene therapy to market and has been expanding its footprint in this biotech hotspot for gene and cell therapy ever since, according to Chief Scientific Officer Federico Mingozzi, Ph.D.

Headquartered in Philadelphia, Pennsylvania, Spark Therapeutics is helping the city become a premier spot for cell and gene therapy.

In 2013, Spark Therapeutics was founded as a result of the technological and scientific know-how developed at the Childrens Hospital of Philadelphia (CHOP). Now part of the Roche Group, the company is committed to further growing Philadelphia as a global hub of cell and gene therapy. With ambitious growth plans, the company remains focused on extending its scientific platform and unique competencies to evaluate and select a portfolio of gene therapies targeting diseases of the retina, hemophilia A, lysosomal storage disorders, and neurodegenerative diseases. They currently have four programs in clinical trials advancing therapies to treat hemophilia A, Pompe disease, and choroideremia.

Chief Scientific Officer Federico Mingozzi, Ph.D.

With Roches worldwide reach and resources, we are closer than weve ever been before to fulfilling our vision of living in a world where no life is limited by genetic disease, Mingozzi says. Although many gene therapy studies focus on genetic diseases, Mingozzi notes, there is an appetite to expand gene therapy to more common indications.

Despite the challenges of 2020, there has been tremendous growth at Spark. The company now employs more than 500 people and has a growing footprint in West Philadelphia. Spark has hired more than 130 employees since the start of 2020 and does not intend to slow down as the company heads into the upcoming year. Part of that expansion brought Senior Research Associate Phillip Price, Ph.D. to Spark in February. Drawn by the companys passion and commitment to treating genetic diseases, Price recalls thinking, I knew I wanted to be part of this.

At the time he was hired by Spark, Price had recently completed his doctorate in neuroscience at Emory University and was serving as a visiting researcher at the Mayo Clinic in Jacksonville, Florida. His strong conviction about the need to bring more treatments to more patients living with genetic disease, in tandem with a seamless interviewing process, propelled Price to make the leap from Florida to Pennsylvania.

Senior Research Associate Phillip Price, Ph.D.

Despite working virtually, Price says he was able to get plugged into the company quickly and find a strong relationship between the employer and the employees, who encouraged both personal and professional growth. Price mentions that the company also made significant efforts to understand and respond to the changing dynamics resulting from the pandemic, such as working remotely. He has benefitted from the strong lines of communication from Sparks leadership andits unique employee programs such as half days every other Friday, which have enabled him to step away from work and relax while learning more about Philadelphia.

Although Price experienced some hesitancy in transitioning from academia to industry, that disappeared when he found that throughout the Spark workforce people are dedicated to the learning, listening, and innovation needed to bring the best treatments to patients. Price appreciates that high level of motivation. Gene therapy is not just about therapeutic targets of the future, he notes. This is here now, and the door is open! At Spark, he finds even more excitement since being a part of Roche provides a platform to reach globally.

With experience in studying spinal muscular atrophy, an inherited disorder, Price fits right in with Sparks central nervous system (CNS) team, which has treatments in its pipeline for other genetic diseases, including Huntingtons disease and Batten disease. Already, he describes the environment as very dynamic and based on beneficial processes that help cultivate ideas. Plus, he quickly felt like part of the team, where he says, everyone has a voice.

This is the hope: Philadelphia will continue to attract and grow the community of biotechnology experts and become one of the most important hubs for gene and cell therapy in the world.

The thriving life sciences industry in Philadelphia also appealed to Price. More than 42,000 Philadelphians work in life sciences and the city is now rated the fifth largest research and development (R&D) hub in the nation, boasting $10.5 billion spent annually in R&D, according to statistics provided by Life Sciences Pennsylvania. Plus, international information firm EqualOcean reports, More than 700 life science companies have chosen to come to Philadelphia to develop their products.

In addition to CHOP, the region is home to a number of other world-class life sciences institutions, including the University of Pennsylvania Perelman School of Medicine, Temple University, and the Drexel University College of Medicine. The combination of Philadelphias academic institutions with the thriving environment it provides for biotech start-ups has resulted in an excellent system for networking and collaborating, according to Price.

Assay development scientist Stephanie Kutza at work in Sparks labs.

Mingozzi agrees. He added that Spark is one of a cluster of many active companies in gene and cell therapy that are creating a vibrant biotech center in the city. Having a hub attracts talent from other cities and retains the talent that is already here, Mingozzi says. For example, he notes that Philadelphia is a place where people come for training and then stay for work. Overall, its an incredible success story for Philadelphia, and the field of gene and cell therapy is driving this success, he says.

Still, Mingozzi sees this as just the beginning for Philadelphia. Youll see that the driver of this hub will be a couple of companies, like Spark, and that people trained in Philadelphia will start new companies here, he says. Then gene therapy will become a major contributor for the local economy.

This diverse city, however, offers much more than science. Price lives downtown, which he describes as a very lively and inspiring atmosphere. As a history buff, he enjoys exploring Philadelphia with its many important sites, including Independence Hall, the Liberty Bell and the Betsy Ross House. Thinking of the architecture, museums, and parks, Price has nothing but appreciation for the City of Brotherly Love, saying its really quite beautiful here.

Notwithstanding its beauty, even more biotech growth lies ahead in Philadelphias future. As Mingozzi sees it, This is the hope: Philadelphia will continue to attract and grow the community of biotechnology experts and become one of the most important hubs for gene and cell therapy in the world.

Spark ranks among the top employers in Science Careers 2020 Top Employer survey. Read more

See the article here:
The City of Biotech Love - Science Magazine

Roger Perlmutter has a few more moves to make before he exits the top job in Merck R&D.

The pharma giant is buying VelosBio for $2.75 billion in cash, grabbing a promising ROR1 antibody drug conjugate that is now in Phase I and Phase II studies for hematologic malignancies and solid tumors.

Perlmutter, who will leave at the end of the year after building Keytruda into a monster drug franchise, clearly likes the data hes seen from the biotech. And the oncology group sees plenty of opportunities for the rest of the pipeline at VelosBio, which raised a crossover round of $137 million just a few months ago in an apparent move toward an IPO.

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Citing pandemic, Sarepta defers much-anticipated DMD gene therapy PhIII, will head to regulators after 10-person study - Endpoints News

MALVERN, Pa., Nov. 06, 2020 (GLOBE NEWSWIRE) --Ocugen, Inc.. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing and commercializing transformative therapies to cure blindness diseases, today reported third quarter 2020 financial results along with a general business update.

I am proud of the advancements we have made this quarter in developing our modifier gene therapy and novel biologic product candidates. We remain on-track to achieve multiple near and mid-term milestones with a plan to initiate four Phase 1/2a trials during 2021 and 2022. Additionally, I am pleased that we have raised approximately $28million of gross proceeds in 2020, which have extended our cash runway for continued development of our product candidates, said Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen.

Business Highlights:

Third Quarter 2020 Financial Results:

About Ocugen, Inc.

Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. For more information, please visit http://www.ocugen.com.

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Corporate Contact:Ocugen, Inc.Sanjay SubramanianChief Financial OfficerIR@Ocugen.com

Media Contact:LaVoieHealthScienceKatie Gallagherkgallagher@lavoiehealthscience.com+1 617-792-3937

(tables to follow)

OCUGEN, INC.CONSOLIDATED BALANCE SHEETS(UNAUDITED)

OCUGEN, INC.CONSOLIDATED STATEMENTS OF OPERATIONS(UNAUDITED)

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Ocugen Provides Business Update and Third Quarter 2020 Financial ResultsFirst Gene Therapy Candidate OCU400 On Track to Enter the Clinic in 2021 -...

Is your team confident in handling the complicated logistics that come with cell and gene trials?

TORONTO (PRWEB) November 06, 2020

Nothing is standard about the study design, study conduct or the regulatory process in cell and gene therapy trials. Every cell and gene therapy is different due to the specific characteristics of each product, with a complicated regulatory approval process and unique therapeutic challenges.

Do you have confidence in your trials endpoints? Are you pursuing an accelerated pathway or special designation? Will you need to plan for a centralized site model or remote monitoring? Is your team confident in handling the complicated logistics that come with cell and gene trials?

In this webinar, participants will learn an end-to-end approach to bringing a cell and gene therapy to market. You will receive advice from experts responsible for supporting over 100 advanced cell and gene therapy programs. They will share each step of this process covering the planning, execution, submission and post-marketing for trials in the US and EU. The information provided will help participants determine their next step in the development of their cell and gene therapy product.

Join Ewan Campbell, Advanced Therapy & Biotech Director, Veristat; Kevin Hennegan, Senior Regulatory Strategist, Veristat; and Rachel Smith, Project Director, Veristat in a live webinar on Friday, November 20, 2020 at 11am EST (4pm GMT/UK).

For more information, or to register for this event, visit Roadmap to Market for Cell and Gene Therapies.

ABOUT XTALKS

Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year, thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

To learn more about Xtalks visit http://xtalks.comFor information about hosting a webinar visit http://xtalks.com/why-host-a-webinar/

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Roadmap to Market for Cell and Gene Therapies, Upcoming Webinar Hosted by Xtalks - PR Web

This webcast features:Leisha Kopp, Applications Scientist, Mirus Bio

Cell and gene therapies show unparalleled promise to improve the human condition by eradicating cancer and rectifying genetic disorders. However, low viral titers and capacity constraints in viral vector manufacturing processes can dramatically hinder the progress of these transformative therapies.

TheTransIT-VirusGEN Transfection Reagent from Mirus Bio offers a simplified, cost-effective workflow for recombinant lentivirus (LV) and adenoassociated virus (AAV) generation.TransIT-VirusGEN Transfection Reagent is also effective in both adherent and suspension HEK 293derived cell types, making it the superior reagent choice for most large-scale production platforms. In addition, Mirus developed LV and AAV enhancers to increase functional virus titers two to three fold over previously optimized conditions and offer up to 10-fold titer improvements over polyethylenimine (PEI)-based formulations.

Just fill out the form below to watch the recorded webcast now.

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Accelerate Cell and Gene Therapy Development and Increase Manufacturing Capacity with Higher Titer LV and AAV Transfection Platforms - BioProcess...

Homology Webcast / Conference Call Scheduled for Friday, November 6 at 4:30 p.m. ET

BEDFORD, Mass., Nov. 05, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the upcoming oral presentation of data from the dose-escalation portion of the Companys ongoing Phase 1/2 pheNIX gene therapy clinical trial with HMI-102 for adults with phenylketonuria (PKU), the worlds first PKU gene therapy clinical trial. The data will be presented by pheNIX principal investigator Olaf Bodamer, M.D., Ph.D., FACMG, FAAP, Park Gerald Chair in Genetics & Genomics and Associate Chief of Genetics & Genomics at Boston Childrens Hospital, a premier center for metabolic disorders and the first site to enroll a patient in the pheNIX trial. The pheNIX trial results will be featured at the virtual New England Consortium of Metabolic Programs (NECMP) annual meeting, which is focused on new research in metabolic disorders, including PKU, on Friday, November 6. NECMP includes metabolic clinics, healthcare providers, patient organizations and others dedicated to increasing knowledge of metabolic disorders and improving delivery of healthcare to patients.

We are pleased that additional data from our pheNIX trial, the first-ever gene therapy trial for PKU, will be shared for the first time by a respected leader in the field of metabolic disorders and directly with the PKU community as part of a featured, peer-reviewed scientific session, stated Gabe Cohn, M.D., Chief Medical Officer of Homology Medicines. The oral presentation by Dr. Bodamer will include data from three dose cohorts in the dose-escalation phase of our pheNIX trial, the results of which are informing the upcoming expansion phase of our study.

Webcast/Conference Call The NECMP annual meeting is for members of the Consortium. Homology will host a conference call and webcast following the meeting on Friday, November 6 at 4:30 p.m. ET. The webcast will be accessible on Homologys website in the Investors section, and the webcast replay will be available on the website for 90 days following the presentation. To access using the conference call line, dial (866) 244-8091 (U.S./Canada toll-free) or (602) 563-8623, with Conference ID 7394503.

About HMI-102HMI-102 is an investigational gene therapy in clinical development for the treatment of phenylketonuria (PKU) in adults. HMI-102 is designed to encode the PAH gene, which is mutated in people with PKU, delivered via the liver-tropic AAVHSC15 vector. Homology has received Fast Track Designation and orphan drug designation for HMI-102 from the U.S. Food and Drug Administration (FDA), and orphan drug designation from the European Medicines Agency (EMA).

About Phenylketonuria (PKU)PKU is a rare inborn error of metabolism caused by a mutation in thePAHgene. PKU results in a loss of function of the enzyme phenylalanine hydroxylase, which is responsible for the metabolism of phenylalanine (Phe), an amino acid obtained exclusively from the diet. If left untreated, toxic levels of Phe can accumulate in the blood and result in progressive and severe neurological impairment. Currently, there are no treatment options for PKU that target the underlying genetic cause of the disease. According to the National PKU Alliance, PKU affects nearly 16,500 people in the U.S. with approximately 350 newborns diagnosed each year. The worldwide prevalence of PKU is estimated to be 50,000 people.

About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing for the release of clinical data from the Phase 1/2 pheNIX trial; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarterly period endedJune 30, 2020and our other filings with theSECcould cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

Company ContactsTheresa McNeely Chief Communications Officer and Patient Advocate tmcneely@homologymedicines.com781-301-7277

Media Contact: Cara Mayfield Senior Director, Patient Advocacy and Corporate Communications cmayfield@homologymedicines.com781-691-3510

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Homology Medicines Announces Upcoming Oral Presentation on pheNIX Gene Therapy Clinical Trial for Adults with PKU - GlobeNewswire

CRISPR Therapeutics and Vertex have presented updated data on their CRISPR/Cas9 gene-editing therapy in sickle cell disease and beta thalassemia, showing patients continue to benefit from the drug. Yet, hemoglobin levels fell in two of the seven patients in the latest update, raising early questions about the durability of CTX001.

One year ago, CRISPR and Vertex linked CTX001 to improvements in two hemoglobinopathy patients. The results were a milestone for gene editing, but CTX001 needs to build on the early success if it is to emerge as a viable rival to bluebird bios LentiGlobin gene therapy, which is now set to be submitted for FDA approval in late 2022.

The LentiGlobin delays create an opportunity for CTX001. The question is whether CRISPR and Vertex can take it. Ahead of the American Society of Hematology's (ASH's) annual meeting, the partners shared new data that shed light on the potential prospects of CTX001.

Fetal hemoglobin levels rose in all seven patients after administration of CTX001 and have remained elevated for up to 15 months of follow-up. The five beta thalassemia patients last needed packed red blood cells in the first month or two after receiving CTX001, having previously more than 10 units a year, and neither of the sickle cell patients have suffered vaso-occlusive crises since being treated.

There are potential points of concern in the data, though, particularly given the likelihood of CTX001 facing competition. Hemoglobin fell from 12.5 to 11.6 in one beta thalassemia patient between the fifth and sixth months. The drop followed a decrease from 12.9 to 12.5 between the fourth and fifth months.

Investigators also tracked a decline in hemoglobin levels in one of the sickle cell patients. Levels fell from 11.8 to 10.3 between month nine and month 12.

The declines happened in two of the three patients with the longest follow-up, raising the question of whether similar trends will play out in other subjects in the coming months. Those concerns are countered by data on the first beta thalassemia patient, whose hemoglobin level is higher than ever 15 months after treatment with CTX001.

Safety is another concern. In an update earlier this year, CRISPR and Vertex revealed they had seen seven serious adverse events across three patients, suggesting bluebirds LentiGlobin may have the edge in terms of safety. The ASH update discussed a new patient who suffered four serious adverse events that may be related to CTX001.

Such details could matter, given CTX001 is still likely to cede a head start to LentiGlobin. Yet, CRISPR sees CTX001 as a best-in-class treatment and, with bluebird stumbling, thinks the drug is well placed to capture a sizable slice of the market.

CRSP has mentioned they are working on bridging the development timeline gap (~1-2 yrs now) w/ BLUE, and they expect BLUE's earlier launch and investment in developing+educating the mkt could benefit CRSP w/o loss of a bolus of pts, analysts at Jefferies wrote in a note to investors.

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CRISPR, Vertex show CRISPR/Cas9 gene-editing therapy works in more patients - FierceBiotech

Short-term pulmonary outcomes of these medications are published and longitudinal monitoring is active, they wrote. Most studies have demonstrated favorable responses in overall muscle strength compared to natural history, but the pulmonary outcomes vary.

Studies included in the review revolved around 3 recently approved treatments:

Nusinersen

Although nusinersen has demonstrated efficacy on respiratory muscle function, the effect varies by SMA subtype, according to the researchers. They cited a study that found the treatment had little impact on patients with SMA1 A and B and had better response in patients with SMA1 C when it came to ventilatory and thoracoabdominal patterns.

Onasemnogene abeparvovec-xioi

In the pivotal phase 1 START trial for the gene therapy, survival data was striking, with an event-free survival of 100% without need for permanent ventilation at 20 months compared with the 8% event-free survival in natural history studies. Throughout the study, 83% (n = 10) of patients required at least 1 hospitalization for respiratory illness with increased length of noninvasive ventilation support, although no hospitalizations surpassed 8 days. Patients in the study had 1.4 respiratory-related hospitalizations per year, with a mean length of stay of 6.7 days.

According to the researchers, the study data suggest that overall pulmonary morbidity and consequent healthcare utilization may have decreased, but some patients may still require escalation of respiratory support during illnesses.

Other data, coming from the phase 3 STRIVE trial, the ongoing SPR1NT study, and real-world experience with the treatment in 3 tertiary centers in Ohio, have made the case for improved respiratory and nutritional outcomes with the gene therapy as researchers await long-term data.

Risdiplam

In the multicenter FIREFISH trial, 90% of the 21 patients were alive without permanent ventilation and rarely required hospitalized after 12 months of treatment. After 23 months, 81% were alive without permanent ventilation. All patients were able to swallow safely and most (95%) were able to be fed orally or with supplemental gastrostomy feeds.

Promising data have also emerged from the SUNFISH and JEWELFISH trials, both of which included patients with varying subtypes of SMA. The researchers also highlighted the RAINBOWFISH study, which is actively recruiting patients with SMA type 1. According to the researchers, the results of the study could be transforming to those with a neonatal diagnosis of SMA type 1.

Looking forward

Looking toward the future, the researchers of the review noted that monitoring of short- and long-term outcomes is still critical, as its still too early to determine sustained improvement in pulmonary outcomes. They add that currently available data come from a limited number of patients and emphasize the need for more multicenter trials to better validate the impact of novel treatments.

Reference

Paul G, Gushue C, Kotha K, Shell R. The respiratory impact of novel therapies for spinal muscular atrophy. Pediatr Pulmonol. Published online October 24, 2020. doi:10.1002/ppul.25135

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Researchers Explore Respiratory Impact of Novel Therapies in SMA - AJMC.com Managed Markets Network